7:30 am Registration & Welcome Coffee

8:25 am Chair’s Opening Remarks

8:30 am Lipid-Based Nanocarriers: Where Are We Today?

Synopsis

  • As industry witnesses a diversification how lipid-based delivery vehicles, as new lipids are discovered, payloads loaded and diseases beyond infectious targeted, where is groundbreaking work taking place and laying the foundations for future innovation in drug delivery?
  • An overview of industry’s progress and where further research will be critical to ensuring continued translation of next-generation delivery vehicles to the clinic and towards patients

Beyond the Liver: Achieving Efficacious Extrahepatic Delivery

9:00 am Nucleic Acid Delivery Systems for RNA therapy and Genome Editing

Synopsis

  • Development of new RNA delivery systems to non-hepatic tissue, including lung and HSC’s
  • Advances in circular RNA delivery
  • High throughput development of RNA delivery systems 

9:30 am Development of Safer Lipid Excipients & Efficient LNP Formulations

  • Vicent Nebot Chief Technology Officer and Head of EU, Curapath

Synopsis

  • CMC development overview in novel functional excipients
  • Seamless LNP formulation screening, process development and GMP manufacturing through a vertically integrated platform 
  • Beyond PEGylated lipids: Non-immunogenic shielding lipids in vaccines

10:00 am Speed Networking Break

Synopsis

Meet and connect with your peers in this dedicated networking session

10:45 am The Future of Tissue-Targeted Lipid Nanoparticle Nucleic Acid Delivery

  • Stephan Stern Laboratory Co-Director, Director of Research & Development, Nanotechnology Characterization Laboratory, National Cancer Institute

Synopsis

  • Current marketed lipid nanoparticle-based nucleic acid therapeutics and future tissue targeted applications
  • Nucleic acid lipid nanoparticle formulation and characterization, non-targeted LNP biodistribution, optimizing tissue targeted delivery
  • Examples from: Hematopoietic stem cell-targeted CRISPR LNP for Sickle cell treatment ; Targeted siRNA delivery for pancreatic cancer therapy– in vivo models and translational development ; APC-targeted mRNA LNP vaccines for increased potency and safety

11:15 am Phospholipids in Vaccines

Synopsis

  • Highlighting the safety and efficacy of phospholipids which make them ideal excipients
  • Discuss the composition and purpose of phospholipids in LNP vaccines
  • Phospholipids acting as adjuvants in liposomal vaccines 

11:30 am Panel Discussion: LNP Design Principles: How Does Nanoparticle Design & Delivery Route Influence Extrahepatic Targeting Properties?

Synopsis

  • How to better engineer and select particles with the most attractive properties?
  • Which novel lipid discoveries are showing promise for efficient in vivo delivery of nucleic acid drugs?
  • How can we better characterize lipids and define their properties and chemical identities to facilitate the next-generation development of lipids?
  • What formulation considerations are needed for differing routes of delivery, for instance intramuscular, subcutaneous? 

12:15 pm Lunch Break

Synopsis

Take this chance to meet the expert speakers, connect with your peers and explore our exhibition booths

1:30 pm Extrahepatic Delivery of mRNA Based on Ionizable Cationic Lipid Library: From Hepatocyte to Activated Stellate Cells, from Normal to Disease State

  • Hideyoshi Harashima Professor of Pharmaceutics and the chair of the Laboratory of Molecular Design of Pharmaceutics, Faculty of Pharmaceutical, Hokkaido University

Synopsis

  • From in vivo screening, we have identified a few ionizable cationic lipid such as CL4H6 which can exert silencing activity of siRNA more efficient than MC3 in hepatocytes after intravenous administration
  • CL4H6LNP can be applied for genome editing successfully, we established to formulate CRISPR/Cas9 ribonucleoprotein (RNP) into LNP
  • Then we challenged to find a new system which can target an activated stellate cells (aHSC) which play an important role in the progression of liver fibrosis based on our library of iCL; We have identified CL15A6 which has different hydrophilic head group and hydrophobic lipid tail from CL4H6
  • Another example is an optimized formulation to target splenic B-cells for pDNA, which can induce efficient antitumor effects via enhanced CTL activity
  • These results indicate that a tailored formulation with ionizable cationic lipids library can provide a cell selective targeting of nucleic acids/RNP without ligands for next generation nanomedicine

2:00 pm AI-Assisted Design of Lipid Nanoparticles for Effective Organ Specific Delivery

  • Daniel Quevedo Associate Director, Strategy & Business Development, METiS Therapeutics

Synopsis

  • METiS has developed a proprietary organ-targeting LNP discovery engine that combines AI, Machine Learning and high-throughput in vivo screening
  • The platform iteratively designs and optimizes LNPs that can specifically target various organs
  • This presentation will highlight in vivo data for METiS’ IV-administered lung-targeting

Solidifying Safety for Next-Generation Hepatic Delivery

2:15 pm Delivering Innovative Gene Editing Therapies to The Liver in Addition to Extrahepatic Locations with Optimized Selectivity

Synopsis

  • Combining Gene Writing with innovative delivery unlocks immense value for genetic medicine
  • Tessera is developing a proprietary nonviral platform for in vivo delivery to multiple tissues (hepatic and extrahepatic delivery)
  • Presentation highlighting improved safety and efficacy of cell therapy delivery for hepatic and extrahepatic delivery in vivo

2:45 pm Polymer Lipid Hybrids: A Chemical Toolbox for LNP Formulations

  • Vicent Nebot Chief Technology Officer and Head of EU, Curapath

Synopsis

  • Novel polymer-lipids, beyond PEG
  • Advantages of Polysarcosine PSar
  • Lipid Manufacturing
  • LNP Manufacturing

3:15 pm Poster & Afternoon Networking Session

Synopsis

The learning and networking continues! This is the perfect opportunity to present & discuss your recent work with fellow drug delivery leaders & innovators!

Payloads Navigating Complex Biological Barriers

3:45 pm Interactive Roundable: Enabling New Modalities to Achieve Their Therapeutic Potential

  • Saikat Manna Senior Scientist, Delivery & Formulation - Messenger Ribonucleic Acid Center of Excellence, Sanofi U.S.
  • Manuel sanchez-felix Research Advisor, Novartis

Synopsis

As our understanding of new nucleic-acid modalities improve, and bioavailability assessments reveal a necessitated need for drug delivery vehicles, there is urgent need for lipid-based nanoparticle delivery system development to keep up with pharmaceutical progress to see the therapeutic potential of pipelines reached.

With LNPs established for mRNA / RNA therapies, and new lipid chemistries being discovered, use this roundtable discussion to gain peer-to-peer feedback:

  • Could a greater understanding of barriers related to targeting different tissues allow for a better class of LNPs be developed? Considering route of administration and tissue related barriers
  • What critical carrier attributes need to be addressed in order protect payloads from release when traveling across membranes?

4:45 pm Reaching the Nucleus: Lipid-Based DNA Therapeutics

Synopsis

  • The final frontier in gene delivery: nuclear delivery of DNA. How do LNPs stack up against older platforms?
  • How can we systematically engineer nanoparticles for improved nuclear delivery of DNA?
  • In vivo validation to develop translatable platforms, with rigorous safety examinations supporting clinical translation

5:15 pm Chair’s Closing Remarks & End of Day One

5:20 pm Networking & Drinks Reception